On 29 January, the first such spinal muscular atrophy treatment was performed at the Clinical Center, which a patient under the age of 18 received with social security benefit.
According to the announcement of Professor Miklós Kásler, the Minister of Human Capacities last November, the treatment for SMA, spinal muscular atrophy with the only product registered in Europe (Spinraza) is now available in Hungary without individual licensing. This way, all young people and children under the age of 18 can have access to the most up-to-date gene therapy. Without treatment, SMA can lead to death, but even less tragically, the patients can become completely bedridden.
Spinraza is the first gene therapy treatment, the clinical effectiveness and safety of which have been proven by several clinical trials, this new drug stops the progression of the disease. The product should be administered into the spinal fluid every four months. In Hungary, the University of Pécs is one of the centres authorized to provide the treatment.
Andor Sebestyén, President of the Clinical Center said in connection with the positive news: “Two to three children are diagnosed with SMA every year at the UP Clinical Center Department of Medical Genetics. It gives confidence to the children and their families that they can receive treatment with social security benefit and the families are now relieved of a significant burden due to the fact that they can receive treatment in a centre close to the children's home” - the President emphasized.
The first Spinraza treatment in Pécs was performed at the Department of Neurology, led by dr. Endre Pál associate professor. The patient is a 17-year-old young person suffering from type II of the disease and is consequently a wheelchair user but with the treatment a significant improvement in the quality of life can be achieved. Soon they will provide help for another patient under 18.
Source:
PTE